The Food and Drug Administration (FDA) has granted Orphan Drug designation and Rare Pediatric Disease designation to WVE-210201 (Wave Life Sciences), an investigational treatment for Duchenne muscular dystrophy (DMD).

DMD is predominately caused by out-of-frame deletions in the dystrophin gene, which results in absent or defective dystrophin protein, leading to progressive and irreversible muscle function loss. WVE-210201, a stereopure oligonucleotide, has been shown to induce skipping of exon 51 of dystrophin pre-mRNA in preclinical studies. In vitro experiments of this novel treatment in DMD patient-derived myoblasts showed efficient exon 51 skipping and dystrophin protein restoration.

A Phase 1 study is currently underway and is expected to enroll 40 DMD patients (5–18 years old) with gene mutations amenable to exon 51 skipping to test the safety and tolerability of WVE-210201.

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“Our team is motivated by a sense of urgency and compassion for the patients, families and caregivers affected by Duchenne muscular dystrophy and other serious, life-threatening conditions with high areas of unmet need,” said Michael Panzara, MD, MPH, Neurology Franchise Lead of Wave Life Sciences. “We are very pleased to receive these two important designations from the FDA and believe they further reinforce the potential of WVE-210201 to help boys suffering from DMD.”

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